FDA News Recap: April 2025

Welcome back to our recap of this month's news and updates from the US Food and Drug Administration (FDA)!

April 2025 continued the FDA’s momentum in ushering in transformative therapies, with approvals spanning rare diseases, autoimmune disorders, and hematologic conditions. From the first approved therapy for IgG4-related disease to gene therapy for recessive dystrophic epidermolysis bullosa, the month’s decisions reflect a growing focus on targeted, patient-specific treatments.

In addition, label expansions and regulatory milestones added depth to existing therapeutic arsenals, while delays and a key Complete Response Letter underscored the rigorous standards that shape drug development. Here’s a look at the most significant FDA developments from April 2025.

Want to hear from the experts? Look for our Related Content links underneath blurbs for insight into how this decision might influence practice moving forward.

FDA News Month in Review: April 2025

Atrasentan (Vanrafia) Receives Accelerated Approval in IgAN

On April 2, 2025, Travere Therapeutics announced the FDA granted accelerated approval to atrasentan (Vanrafia) for reducing proteinuria in patients with IgA nephropathy (IgAN). Based on interim results from the phase 3 ALIGN trial, atrasentan demonstrated significant reductions in proteinuria, supporting its use as a disease-modifying therapy in IgAN.​

Related Content: Understanding Atrasentan (Vanrafia) for IgA Nephropathy, with Richard Lafayette, MD

FDA Approves Inebilizumab (Uplizna) as First IgG4-RD Therapy for Adults

On April 3, 2025, Horizon Therapeutics announced the FDA approved inebilizumab (Uplizna) as the first treatment for adults with IgG4-related disease (IgG4-RD). Based on data from the phase 3 NATRON trial, inebilizumab significantly reduced the risk of disease flare compared to placebo, marking a milestone in managing this chronic fibroinflammatory condition.​

FDA Approves Maralixibat (Livmarli) Tablet for Alagille Syndrome, PFIC

On April 14, 2025, Mirum Pharmaceuticals announced the FDA approved maralixibat (Livmarli) tablets for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC). Based on data from the ICONIC and MARCH studies, maralixibat demonstrated significant reductions in pruritus severity and improvements in liver function parameters.​

FDA Approves Label Expansion for Osilodrostat for Cushing’s Syndrome

On April 16, 2025, Recordati Rare Diseases announced the FDA approved a label expansion for osilodrostat (Isturisa) to include the treatment of Cushing's syndrome in pediatric patients aged 6 years and older. Based on data from the phase 2/3 LINC-4 study, osilodrostat effectively normalized cortisol levels and improved clinical symptoms in this population.​

FDA Updates Mavacamten Label in Obstructive Hypertrophic Cardiomyopathy

On April 17, 2025, Bristol Myers Squibb announced the FDA approved an updated label for mavacamten (Camzyos) to include long-term safety and efficacy data in patients with obstructive hypertrophic cardiomyopathy (oHCM). Based on findings from the EXPLORER-HCM and VALOR-HCM trials, mavacamten demonstrated sustained improvements in functional capacity and symptom burden over extended treatment periods.​

FDA Approves Dupilumab (Dupixent) for Chronic Spontaneous Urticaria

On April 18, 2025, Regeneron Pharmaceuticals and Sanofi announced the FDA approved dupilumab (Dupixent) for the treatment of chronic spontaneous urticaria (CSU) in patients aged 12 years and older. Based on results from the LIBERTY-CUPID trials, dupilumab significantly reduced itch severity and hives, offering a new therapeutic option for patients with inadequately controlled CSU.​

FDA Issues Complete Response Letter to Aflibercept 8 mg for Extended Dosing

On April 18, 2025, Regeneron Pharmaceuticals announced the FDA issued a Complete Response Letter (CRL) for aflibercept 8 mg, citing the need for additional data on extended dosing intervals in neovascular age-related macular degeneration (nAMD). The CRL did not raise concerns about safety or efficacy but requested further information to support the proposed dosing regimen.​

FDA Approves PZ-cel Gene Therapy for Recessive Dystrophic Epidermolysis Bullosa

On April 29, 2025, Krystal Biotech announced the FDA approved beremagene geperpavec (Vyjuvek) as the first gene therapy for the treatment of recessive dystrophic epidermolysis bullosa (RDEB). Based on data from the GEM-3 trial, Vyjuvek demonstrated significant improvements in wound healing and skin integrity in patients with RDEB.​

FDA Approves Upadacitinib, Expanding Treatment for Adults With Giant Cell Arteritis

On April 29, 2025, AbbVie announced the FDA approved upadacitinib (Rinvoq) for the treatment of giant cell arteritis (GCA) in adults. Based on results from the SELECT-GCA trial, upadacitinib achieved sustained remission and reduced glucocorticoid use, providing a novel oral treatment option for GCA.​

FDA Delays Decision on Elamipretide for Barth Syndrome

On April 30, 2025, Stealth BioTherapeutics announced the FDA delayed its decision on elamipretide for the treatment of Barth syndrome, requesting additional data to support the New Drug Application (NDA). The delay follows the agency's request for further evidence of clinical benefit in this ultra-rare mitochondrial disorder.​

FDA Approves Nipocalimab for Generalized Myasthenia Gravis in Adults, Children

On April 30, 2025, Johnson & Johnson announced the FDA approved nipocalimab (Zilbrysq) for the treatment of generalized myasthenia gravis (gMG) in adults and children aged 12 years and older. Based on data from the Vivacity-MG study, nipocalimab significantly improved muscle strength and reduced disease severity in patients with gMG.​